Healthy VolunteersPhase 1NCT06324604
Safety, Pharmacokinetics, and Pharmacodynamics of MTX-101 in Healthy Adults and Patients
A Phase 1 study testing MTX-101 in people with Healthy Volunteers and Type 1 Diabetes, run by Mozart Therapeutics Australia Pty Ltd at 3 sites in Australia.
Trial summary
First in human study to understand the potential side effects of MTX-101, how long MTX-101 lasts in the human body, and how MTX-101 affects specific human immune cells. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy Volunteers, Type 1 Diabetes
- What is being tested?
- MTX-101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 96 participants
- Where
- 3 sitesAustralia
- Study dates
- 13 Jun 2024to 30 Jun 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Adults, age ≥ 18 and ≤ 65 years at the time of anticipated dosing (Day 1).
- Healthy individuals without known current or chronic medical conditions, including no history of any autoimmune diseases, in the opinion of the Investigator.
- Body mass index (BMI) ≥ 18 kg/m2 and ≤ 35 kg/m2 AND body weight ≥ 55 and ≤ 120 kg.
- Negative Coronavirus Disease 2019 (COVID-19) test within 24 hours prior to each dose.
- Persons of child-bearing potential must have a negative pregnancy test and either abstain from sex or use highly effective method(s) of birth control from Day 1 through the duration of the study.
Who cannot join
- Clinically significant findings in physical examination (PE), vital signs (blood pressure, heart rate, and body temperature), electrocardiogram (ECG), and safety laboratory parameters at Screening in the opinion of the Investigator.
- Prior or concurrent malignancies.
- Renal function calculated by the Chronic Kidney Disease-Epidemiology (CKD-EPI) equation with estimated glomerular filtration rate (eGFR) \< 90 mL/min/1.73 m2 or abnormal level of proteinuria detected by dipstick at the time of Screening.
- Any disease or condition that, in the opinion of the Investigator, might significantly compromise the cardiovascular, hematological, renal, hepatic, pulmonary (including chronic asthma), endocrine (e.g., diabetes), central nervous, or gastrointestinal (including an ulcer) systems.
- Receipt of an investigational drug within 28 days or 5 half-lives (whichever is longer) of the investigational drug(s) prior to Day 1.
- Positive serology for human immunodeficiency virus (HIV) type 1 or 2, hepatitis (Hep) B surface antigen, or Hep C.
Show all 14Show fewer
- Positive test results for drug screen, including alcohol, at the time of Screening or on Day 1 prior to randomization.
- Use of tobacco or nicotine-containing products more than the equivalent of 5 cigarettes/week within 30 days prior to (first) dosing.
- Participants must abstain from nicotine use while inpatient.
- History of receiving a live vaccine within 1 month of Screening.
- History of splenectomy.
- History of COVID or influenza vaccine within 2 weeks prior to Screening.
- Planning to receive any vaccinations during the study period.
- History of recurrent infections of uncertain cause.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Australia3 sites, 3 recruiting
- Recruiting
St Vincent's Hospital Melbourne (SVHM)
Fitzroy, 3065
David O'Neal, MD
- Recruiting
Austin Health
Heidelberg, Victoria, 3084
Elif Ekinci, MD
- Recruiting
The Royal Melbourne Hospital
Melbourne, Victoria, 3050
John Wentworth, MD
About this study
First in human study to understand the potential side effects of MTX-101, how long MTX-101 lasts in the human body, and how MTX-101 affects specific human immune cells.
Read the detailed description
A Phase 1, Part A -prospective, randomized, single-blind, placebo-controlled, single ascending dose (SAD) and multiple ascending dose (MAD) study to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of MTX-101 in healthy adults (HA). Part B - A multi-center, randomized, open-label study to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of MTX-101 in participants with type 1 diabetes (T1D).
- Conditions
- Healthy Volunteers, Type 1 Diabetes
- Being tested
- Placebo, MTX-101
- Keywords
- Healthy Volunteers, Type 1 Diabetes
- Medical subject headings
- Diabetes Mellitus, Type 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
MTX-101
MTX-101Drug
MTX-101 (bispecific CD8 Treg modulator)
| Study group | Role | What they receive |
|---|---|---|
| Cohort AS1 - Healthy Volunteers | Placebo comparator | (n = 6): MTX-101, Dose level 1 IV or Placebo IV, Single dose Receives: Drug: Placebo; Drug: MTX-101 |
| Cohort AS2 - Healthy Volunteers | Placebo comparator | (n = 6): MTX-101, Dose Level 2 IV or Placebo IV, Single dose Receives: Drug: Placebo; Drug: MTX-101 |
| Cohort AS3 - Healthy Vounteers | Placebo comparator | (n = 6): MTX-101, Dose Level 3 IV or Placebo IV, single dose Receives: Drug: Placebo; Drug: MTX-101 |
| Cohort AS4 - Healthy Volunteers | Placebo comparator | (n =6): MTX-101, Dose Level 4 IV or Placebo IV, single dose Receives: Drug: Placebo; Drug: MTX-101 |
| Cohort AS5 - Healthy Volunteers | Placebo comparator | (n = 6): MTX-101, Dose level 6 IV or Placebo IV, Single Dose Receives: Drug: Placebo; Drug: MTX-101 |
| Cohort AM1 - Healthy Volunteers | Placebo comparator | Cohort AM1 (n = 6): MTX-101, Dose Level 5 IV or Placebo IV, dosed on Days 1 and 22 for a total of 2 doses Receives: Drug: Placebo; Drug: MTX-101 |
| Cohort B8 - Type 1 Diabetes Patients | Placebo comparator | * Cohort B8 (n=12):
* MTX-101 up to Dose Group 4 IV Day 1 and 29 Receives: Drug: MTX-101 |
| Cohort B9 - Type 1 Diabetes Patients | Experimental | Optional Cohort B9 (n=12):
• MTX-101 up to Dose 6 IV Day 1 and 29 Receives: Drug: MTX-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of single, ascending dose levels of MTX-101
Assess the safety of single, ascending dose levels of MTX-101 by evaluating the incidence, severity, and seriousness of treatment-emergent adverse events
Measured: Enrollment to 8 weeks post dose
Safety of multiple, ascending dose levels of MTX-101
Assess the safety of multiple, ascending dose levels of MTX-101by evaluating the incidence, severity, and seriousness of treatment-emergent adverse events
Measured: Enrollment to 11 weeks following the last dose
Secondary outcomes (4)
pharmacokinetics (PK) of MTX-101
Characterize the pharmacokinetics (PK) of MTX-101 by measuring the maximum time of occurrence for maximum plasma drug concentration (Cmax)
Measured: Enrollment to 11 weeks following the last dose
pharmacokinetics (PK) of MTX-101
Characterize the pharmacokinetics (PK) of MTX-101 by measuring the time of occurrence for maximum plasma drug concentration (Tmax).
Measured: Enrollment to 11 weeks following the last dose
pharmacokinetics (PK) of MTX-101
Characterize the pharmacokinetics (PK) of MTX-101 by measuring the maximum plasma drug concentration (Cmax), minimum plasma drug concentration (Cmin), and area under the plasma drug concentration versus time curve from time 0 to last measurable concentration (AUC(0-t))
Measured: Enrollment to 11 weeks following the last dose
anti-drug antibody (ADA) formation
Evaluate incidence of anti-drug antibody (ADA) formation by measuring the detect the presence of anti-MTX-101 antibodies in participant's blood.
Measured: Enrollment to 11 weeks following the last dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2024
- Study start13 Jun 2024
- Primary completion31 Mar 2027 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Healthy VolunteersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- Part A will enroll Healthy Adult Volunteers will be randomized, double-blind, placebo-controlled, single ascending dose (SAD), randomized to MTX-101 or placebo in a 1:1 ratio for the first 2 participants (sentinel dosing) and a 3:1 ratio thereafter. Participants in the multiple ascending dose (MAD) will be randomized in a 3:1 ratio and be dosed on Days 1 and 22. Part B of this study plans to enroll at least 24 (up to 44) participants with CeD or T1D, randomized in a 1:1 ratio, in 2 sequential cohorts. Approximately 12 CeD and 12 T1D patients will be enrolled in Part B, with the option to enroll up to a maximum of 44 total (T1D patients). Participants in Cohort B8 will be dosed with MTX-101 on Days 1 \& 29. Participants in Cohort B9 will be dosed with MTX-101 or placebo on Day 1 and MTX-101 on Day 29. Patients will be followed for 6 months after the first dose.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 96 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mozart Therapeutics Australia Pty Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06324604
- Sponsor's protocol ID
- MT-101-101
- Status last verified
- May 2026
- First submitted
- 27 Feb 2024
- Last updated
- 18 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety, Pharmacokinetics, and Pharmacodynamics of MTX-101 in Healthy Adults and Patients
- ClinicalTrials.gov ID
- NCT06324604
- Acronym / sponsor ID
- MT-101-101
- Conditions
- Healthy Volunteers, Type 1 Diabetes
- Keywords
- Healthy Volunteers, Type 1 Diabetes
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 96 participants (estimated)
- Lead sponsor
- Mozart Therapeutics Australia Pty LtdIndustry (pharma, biotech, devices)
- Study start
- 13 Jun 2024 (actual)
- Primary completion
- 31 Mar 2027 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 22 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
First in human study to understand the potential side effects of MTX-101, how long MTX-101 lasts in the human body, and how MTX-101 affects specific human immune cells.
Detailed description
A Phase 1, Part A -prospective, randomized, single-blind, placebo-controlled, single ascending dose (SAD) and multiple ascending dose (MAD) study to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of MTX-101 in healthy adults (HA). Part B - A multi-center, randomized, open-label study to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of MTX-101 in participants with type 1 diabetes (T1D).
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 8
- Model description
- Part A will enroll Healthy Adult Volunteers will be randomized, double-blind, placebo-controlled, single ascending dose (SAD), randomized to MTX-101 or placebo in a 1:1 ratio for the first 2 participants (sentinel dosing) and a 3:1 ratio thereafter. Participants in the multiple ascending dose (MAD) will be randomized in a 3:1 ratio and be dosed on Days 1 and 22. Part B of this study plans to enroll at least 24 (up to 44) participants with CeD or T1D, randomized in a 1:1 ratio, in 2 sequential cohorts. Approximately 12 CeD and 12 T1D patients will be enrolled in Part B, with the option to enroll up to a maximum of 44 total (T1D patients). Participants in Cohort B8 will be dosed with MTX-101 on Days 1 \& 29. Participants in Cohort B9 will be dosed with MTX-101 or placebo on Day 1 and MTX-101 on Day 29. Patients will be followed for 6 months after the first dose.
| Arm | Type | Receives |
|---|---|---|
| Cohort AS1 - Healthy Volunteers | Placebo comparator | Drug: Placebo, Drug: MTX-101(n = 6): MTX-101, Dose level 1 IV or Placebo IV, Single dose |
| Cohort AS2 - Healthy Volunteers | Placebo comparator | Drug: Placebo, Drug: MTX-101(n = 6): MTX-101, Dose Level 2 IV or Placebo IV, Single dose |
| Cohort AS3 - Healthy Vounteers | Placebo comparator | Drug: Placebo, Drug: MTX-101(n = 6): MTX-101, Dose Level 3 IV or Placebo IV, single dose |
| Cohort AS4 - Healthy Volunteers | Placebo comparator | Drug: Placebo, Drug: MTX-101(n =6): MTX-101, Dose Level 4 IV or Placebo IV, single dose |
| Cohort AS5 - Healthy Volunteers | Placebo comparator | Drug: Placebo, Drug: MTX-101(n = 6): MTX-101, Dose level 6 IV or Placebo IV, Single Dose |
| Cohort AM1 - Healthy Volunteers | Placebo comparator | Drug: Placebo, Drug: MTX-101Cohort AM1 (n = 6): MTX-101, Dose Level 5 IV or Placebo IV, dosed on Days 1 and 22 for a total of 2 doses |
| Cohort B8 - Type 1 Diabetes Patients | Placebo comparator | Drug: MTX-101* Cohort B8 (n=12): * MTX-101 up to Dose Group 4 IV Day 1 and 29 |
| Cohort B9 - Type 1 Diabetes Patients | Experimental | Drug: MTX-101Optional Cohort B9 (n=12): • MTX-101 up to Dose 6 IV Day 1 and 29 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Adults, age ≥ 18 and ≤ 65 years at the time of anticipated dosing (Day 1).
- Healthy individuals without known current or chronic medical conditions, including no history of any autoimmune diseases, in the opinion of the Investigator.
- Body mass index (BMI) ≥ 18 kg/m2 and ≤ 35 kg/m2 AND body weight ≥ 55 and ≤ 120 kg.
- Negative Coronavirus Disease 2019 (COVID-19) test within 24 hours prior to each dose.
- Persons of child-bearing potential must have a negative pregnancy test and either abstain from sex or use highly effective method(s) of birth control from Day 1 through the duration of the study.
Key exclusion criteria (14 listed)
- Clinically significant findings in physical examination (PE), vital signs (blood pressure, heart rate, and body temperature), electrocardiogram (ECG), and safety laboratory parameters at Screening in the opinion of the Investigator.
- Prior or concurrent malignancies.
- Renal function calculated by the Chronic Kidney Disease-Epidemiology (CKD-EPI) equation with estimated glomerular filtration rate (eGFR) \< 90 mL/min/1.73 m2 or abnormal level of proteinuria detected by dipstick at the time of Screening.
- Any disease or condition that, in the opinion of the Investigator, might significantly compromise the cardiovascular, hematological, renal, hepatic, pulmonary (including chronic asthma), endocrine (e.g., diabetes), central nervous, or gastrointestinal (including an ulcer) systems.
- Receipt of an investigational drug within 28 days or 5 half-lives (whichever is longer) of the investigational drug(s) prior to Day 1.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PlaceboMTX-101 | Drug | Cohort AM1 - Healthy Volunteers, Cohort AS1 - Healthy Volunteers, Cohort AS2 - Healthy Volunteers, Cohort AS3 - Healthy Vounteers, Cohort AS4 - Healthy Volunteers, Cohort AS5 - Healthy Volunteers |
| MTX-101MTX-101 (bispecific CD8 Treg modulator) | Drug | Cohort AM1 - Healthy Volunteers, Cohort AS1 - Healthy Volunteers, Cohort AS2 - Healthy Volunteers, Cohort AS3 - Healthy Vounteers, Cohort AS4 - Healthy Volunteers, Cohort AS5 - Healthy Volunteers, Cohort B8 - Type 1 Diabetes Patients, Cohort B9 - Type 1 Diabetes Patients |
Primary outcome
Safety of single, ascending dose levels of MTX-101
Time frame: Enrollment to 8 weeks post dose
Assess the safety of single, ascending dose levels of MTX-101 by evaluating the incidence, severity, and seriousness of treatment-emergent adverse events
Safety of multiple, ascending dose levels of MTX-101
Time frame: Enrollment to 11 weeks following the last dose
Assess the safety of multiple, ascending dose levels of MTX-101by evaluating the incidence, severity, and seriousness of treatment-emergent adverse events
4 secondary outcomes
pharmacokinetics (PK) of MTX-101
Time frame: Enrollment to 11 weeks following the last dose
pharmacokinetics (PK) of MTX-101
Time frame: Enrollment to 11 weeks following the last dose
pharmacokinetics (PK) of MTX-101
Time frame: Enrollment to 11 weeks following the last dose
anti-drug antibody (ADA) formation
Time frame: Enrollment to 11 weeks following the last dose
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2026.
- Individual participant dataSharing planned: Undecided
Related trials for Healthy Volunteers
Not yet recruiting
Neuroergonomic Analysis of a Brain-Computer Interface Device (BCI)
Healthy Volunteers - Male and Female
RecruitingPhase 1
First-in-Human Trial to Assess REGN20423 in Healthy Adult Participants and Adult Participants With Atopic Dermatitis
Healthy Volunteer, Atopic Dermatitis
Not yet recruitingObservational study
Establishment of Biobank of Nasal Epithelium Samples From Healthy Volunteers
Healthy
RecruitingObservational study
Improved Methods of Cell Selection for Bone Marrow Transplant Alternatives
Graft-Versus-Host Disease, Graft-versus-leukemia
Not yet recruitingObservational study
Screening Protocol
Pain
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MTX-101 in people with Healthy Volunteers and Type 1 Diabetes, run by Mozart Therapeutics Australia Pty Ltd at 3 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Heather Director, Clinical Operations (1-253-358-9586, hwroe@mozart-tx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of single, ascending dose levels of MTX-101 (time frame: Enrollment to 8 weeks post dose). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2026.